Core--Vector

描述(由申请人提供) 病毒载体核心设施是UNC基因的一个组成部分 治疗中心。它已经运行了7年多，并保持了 在这段时间内，产量呈指数级增长。这已经是 通过快速融入新技术发展来实现，因为它们 并通过承诺使这些收益 技术尽可能广泛地为研究人员所用。追求 这些目标将在拟议的研究计划中继续下去。项目1和 2特别说明了 核心和附属研究人员。对于项目1，向量核心将是 负责突变体(双链载体)的大规模生产 和嵌合的和血清型特异的rAAV载体，从而促进 这些试剂的表征。提供新功能的载体将 然后被添加到当前可从向量核心获得的服务列表 (例如，类型I特定载体)。在项目2中，新的方法 慢病毒产生细胞系的建立保证了更大的安全性和 可预测性。然后，我们将把这些技术发展成常规服务 提供给缺乏设施或专业知识的调查人员 这些宝贵的资源(例如项目3和4)。获取专业知识 对于项目3和4来说，在提供 在体内进行实验所需的大量病毒载体。 因此，我们可以防止在概念上简单但在技术上要求高的方面 这些建议不会成为其成功的严重障碍。除了 常规输出的载体材料，训练有素的人的可用性 向量核心的人员为这项研究提供了额外的好处 程序。虽然不在这个PPG的范围内，但向量核心也是 负责人工应用程序的维护和操作 实验室，确保基本的媒介技术转化为实施 当临床媒介试验合理时(即新的硫酸肝素(HS)) AAV2型的纯化)。这将确保生产和检测 病毒式传播。

DESCRIPTION (provided by applicant) The Viral Vector Core Facility is an integral component of the UNC Gene Therapy Center. It has been in operation for over 7 years and has maintained an exponential level of production growth over this time. This has been accomplished by rapid incorporation of new technological developments as they become available, and by a commitment to making the proceeds of these technologies as widely accessible to researchers as possible. The pursuit of these goals will continue within the proposed research program. Projects 1 and 2 are particularly illustrative of the reciprocal interactions between the core and affiliated researchers. For Project 1, the Vector Core will be responsible for the large-scale production of mutant (double stranded vectors) and chimeric and serotype specific rAAV vectors, thus facilitating the characterization of these reagents. Vectors that offer new capabilities will then be added to the list of services currently available from the vector core (e.g. type I specific vectors). In Project 2, new methods for the establishment of lentivirus producer cell lines promise greater safety and predictability. We will then develop these techniques into routine services to be offered to investigators who lack the facilities or expertise to create these invaluable resources (e.g. projects 3 and 4). Access to the expertise of the Vector Core will be critical to projects 3 and 4 in supplying the prodigious quantities of viral vectors needed for experimentation in vivo. Thus, we can prevent a conceptually simple but technically demanding aspect of these proposals from becoming a serious obstacle to their success. Apart from the routine output of vector material, the availability of the highly trained personnel of the Vector Core provides additional benefits to this research program. Although not within the scope of this PPG, the Vector Core is also responsible for the maintenance and operations of the Human Applications Laboratory, ensuring that basic vector techniques translate to implementation when clinical vector trials are justified (i.e. new Haparin sulfate (HS) purification of AAV type 2). This will ensure the production and assay of viral.